New pill aims to reverse brain aging and dementia progression, Phase 1 trial planned in Australia

A new pill, RTR242, being developed by Retro Biosciences, is designed to revive the brain’s natural recycling process to clear toxic protein build-up linked to dementia and other neurodegenerative diseases. The company says the treatment could reverse brain aging by about a decade and plans a Phase 1 trial in Australia, with the first patient to receive the medicine by the end of 2025.

The medicine targets autophagy, the cellular housekeeping process that clears damaged components such as tau and amyloid beta. In many neurodegenerative diseases, autophagy slows or becomes overwhelmed, allowing toxic clumps to accumulate and damage neurons. RTR242 is described as reactivating autophagy so that sick but still-alive brain cells can clear the waste and potentially regain function. Unlike some Alzheimer’s drugs approved or in use that aim to remove plaque to slow decline, Retro’s approach seeks to reset biology toward a younger state by restoring the cell’s cleanup system.

The company has not released preclinical data for RTR242 publicly. It says the pill acts by kickstarting autophagy in cells that are alive but dysfunctional because they are clogged with misfolded proteins. By restarting the process, the therapy could prevent neuron death and restore function, potentially addressing diseases such as Alzheimer’s, Lewy body dementia, and Parkinson’s, which involve toxic proteins that can disrupt autophagy.

Retro Biosciences’ founder and CEO, Joe Betts-LaCroix, has framed the work as part of a broader effort to extend healthy lifespan, rather than merely extending life expectancy. He told Business Insider that adding 10 years of healthy life could be a major milestone, with the company pursuing a Series A funding round of about $1 billion to enable larger studies. The trial’s success would determine the ability to pursue that goal and place Retro among longevity-focused peers like Altos Labs, which has drawn substantial funding from tech investors.

The health landscape for dementia remains daunting: about 7 million Americans live with Alzheimer’s disease, and that number is projected to rise to nearly 13 million by 2050. Parkinson’s disease affects roughly 1 million Americans today, with about 90,000 new cases annually and projections to about 1.2 million by 2030. High-profile dementia diagnoses have drawn public attention to these conditions, including Wendy Williams’ frontotemporal dementia diagnosis in 2023 and Bruce Willis’ announcement that he also has frontotemporal dementia in the same year.

If successful, RTR242 would mark a departure from treatments that seek to slow decline by removing a single plaque, offering instead a therapy that aims to reset cellular aging and restore function. The Australian phase 1 trial would set the stage for larger studies if initial results are positive, which Retro says would bolster its bid for substantial funding and commercial development.